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Cognitive and Neurological Pathologies in Pompe Disease
NCT04639336 · View on ClinicalTrials.gov ↗
Study Summary
The purpose of this study is to better understand the long-term health effects of Pompe disease and to determine if there are any abnormal changes in the brain and peripheral nerves. Additionally, the investigators will study the relationship between the abnormal changes in brain, nervous system findings, and developmental outcomes. The investigators will collect clinical information from clinic visits as well as assessments such as neuroimaging (magnetic resonance imaging (MRI), magnetic resonance spectroscopy (MRS), and diffusion tensor imaging (DTI)), cognition, academic skills, speech and language function, physical therapy and quantitative muscle ultrasound. Subjects will be in this study for at least 3 years and up to 6 years.
Conditions Studied
Study Locations (1)
North Carolina
- Duke University Medical Center — Durham
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 45 participants |
| Start Date | 2016-08-03 |
| Est. Completion | 2029-03 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04639336
The ClinicalTrials.gov registry entry for NCT04639336 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 45 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Duke University, which has 1,129 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Pompe Disease appearing as the primary indexed condition, and to 0 interventions. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04639336 reports 1 study location spanning 1 distinct geographic area — top geographies include North Carolina. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04639336 about?
NCT04639336 is a clinical study titled "Cognitive and Neurological Pathologies in Pompe Disease". The purpose of this study is to better understand the long-term health effects of Pompe disease and to determine if there are any abnormal changes in the brain and peripheral nerves. Additionally, the investigators will study the relationship between the abnormal changes in brain, nervous system fin...
What is the current status of trial NCT04639336?
This trial is currently recruiting. The enrollment target is 45 participants. The study started on 2016-08-03. Estimated completion is 2029-03.
What conditions does trial NCT04639336 study?
This clinical trial studies the following conditions: Pompe Disease. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
Who is sponsoring clinical trial NCT04639336?
This trial is sponsored by Duke University, which has 1,129 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04639336 being conducted?
This trial has 1 study location across North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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